Clinical trials are scientific experiments in which we start with a hypothesis about what we think will happen, and then collect data to either confirm or reject our hypothesis.
A common hypothesis that we ask late in the course of developing a therapy is if the treatment we are testing will lead to a slowing in disease progression or will improve symptoms of disease as compared to a group that received a placebo.
- If we find that the treatment did slow disease progression, then the trial would be called a positive trial.
- If we find that the treatment did not slow disease progression, then that would be a negative trial.
- Sometimes, we don’t get a clear result; for example, the treatment never reached the brain, or the patients that were studied did not change over time as expected. This is called a failed trial.
At early stages of clinical development of a new therapy, the hypotheses that we are testing may relate to whether the investigational drug is safe and well tolerated, or whether it got into the brain and had a clear biological effect that was expected based on treatment’s mechanism of action, often determined in an animal or other laboratory model.
To date, the vast majority of clinical trials for Alzheimer’s disease, Frontotemporal Degeneration, Progressive Supranuclear Palsy and other disorders have been negative or in some cases failed. However, increasingly there have been positive results in small clinical trials of Alzheimer’s therapies that can remove amyloid plaques.
Below are links to the published results of some of the clinical trials carried out by our program in the past:
Anti-amyloid therapies for Alzheimer’s disease
Therapies for Frontotemporal Dementia